| | | |   | Happy Friday. News today on the FDA adcom’s vote on Bluebird’s gene thearpy, and STAT’s Allison DeAngelis weighs in two high-dollar seed rounds.
- Meghana | | | FDA advisers give Bluebird gene therapy a thumbs up An FDA advisory committee has unanimously recommended the approval of Bluebird Bio’s experimental gene therapy — voting 15-0 that its ability to help children with cerebral adrenoleukodystrophy outweighs a demonstrable risk of causing cancer. The drug, eli-cel, is a one-time treatment for the young boys who inherit the rare neurological disorder. “This treatment isn’t a cure, but it at least gives these boys time until hopefully one day we can come up with something better for them,” one ad comm member said. The panel is part of a two-day meeting evaluating Bluebird’s products: Today, the same group of independent advisers will evaluate the company’s gene therapy for sickle cell disease. Read more. | When is data 'practice-changing?' How many Covid-19 vaccines do we need? And what does it mean when the FDA asks for more time? We cover all that and more this week on “The Readout LOUD,” STAT’s biotech podcast. With the world’s largest cancer conference just concluded, we explain the most important data presented at the meeting, including a blockbuster clinical trial that promises to change the treatment of advanced breast cancer. We also discuss an FDA controversy in the making, a pair of new Covid-19 vaccines, and the frustrating process of finding new treatments for depression. Listen here. | Over a decade later, what’s next for the future of immunotherapy? Since 2011, the FDA has approved over three dozen novel immunotherapy treatments for patients with various forms of cancer. While these therapies have improved outcomes for some patients, there are others whose tumors don’t respond well enough to these medicines. Learn how the next generation and personalization of immunotherapies will help deliver more options for people with cancer | Plush funding for two seed-stage biotechs Oncology startup Ratio Pharmaceuticals launched today with $20 million in seed funding and development deals with Bayer and Lantheus Holdings. It’s a hefty fundraising round, far beyond the average $5 million or $10 million check many biotechs get for their first private investment. That’s because the amount of money going toward biotech seed rounds has more than quadrupled in the last five years, jumping from $425 million in 2016 to $2.1 billion last year, according to Pitchbook data. Much of that growth has been fueled by Silicon Valley VCs eager to begin investing in biotech. Khosla Ventures, the VC firm founded in 2004 by tech entrepreneur Vinod Khosla, participated in a $25 million seed round that Relation Therapeutics announced Thursday. Relation, which is based in London, plans to use machine learning and single-cell analysis to create medicines for diseases that currently don’t have treatment options. Ratio, meanwhile, is developing targeted radiopharmaceuticals to treat cancer. Unlike many seed-stage companies, it already has drugs ready for human testing. It plans to file an investigational new drug application this month. | Drug makers flouting the 340B law In the name of profit, many drugmakers are breaking the law. Specifically, they’re circumventing bipartisan legislation called the 340B drug pricing program, which was designed to help patients with low incomes or those living in rural areas. The 1992 law requires drug companies to offer discounted prices to hospitals that serve these patients. In return, Medicaid and Medicare Part B cover these medicines — which has been quite profitable for drugmakers. But in the past few years, some companies have been restricting their discounts, while still receiving government coverage. This includes major players like AstraZeneca, Eli Lilly, Gilead, Pfizer, and many more. Some have flat-out refused to restore the discounts, and have been referred to HHS’s Office of Inspector General, which will evaluate whether these companies should face penalties for “knowingly and intentionally” overcharging hospitals. “The OIG should impose fines on all companies refusing to restore 340B discounts,” opines Maureen Testoni of the nonprofit 340B Health. “In an industry driven by the bottom line, that may be the only way to convince them to obey the law.” Read more. | More reads - Building the nuclear pore complex. (Science)
- Tempus ties up Eli Lilly collab to bring free genomic testing to lung cancer patients. (FierceBiotech)
- BioNTech to soon start mRNA vaccine factory construction in Rwanda. (Reuters)
| Thanks for reading! Until next week,  | | | |
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